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The main purpose of this pilot study is to examine the outcome of a combined bone marrow and kidney transplant from a partially matched related (haploidentical or "haplo") donor. The aim of the combined transplant is to treat both underlying blood disorders and kidney diseases. Ten people are expected to participate in this study.


More information:, ITHANET Clinical Trials


The study will evaluate the safety and tolerability of ACE-536 in beta-thalassemia intermedia patients with anemia and the ability of ACE-536 to generate an erythroid response in these patients. Eligible patients may have received up to 6 red blood cell transfusions during the previous 12 months (with none during the 8-week period prior to study enrollment). An erythroid response in this study is defined as an increase in hemoglobin of at least 1.5 g/dL from pretreatment baseline values, sustained for at least 14 days, in the absence of a red blood cell transfusion. The expected starts in December 2012 and the expected completion date is November 2014.


More information:, ITHANET Clinical Trials

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This is a non randomized, open label, multi site, single dose, Phase 1/2 study in up to 15 adults with β thalassemia major who receive at least 100 mL/kg/year of packed red blood cells (pRBCs) or ≥8 transfusions of pRBCs per year. The study will evaluate the safety and efficacy of autologous hematopoietic stem cell transplantation (HSCT) using LentiGlobin® BB305 Drug Product (autologous CD34+ hematopoietic stem cells transduced with LentiGlobin® BB305 lentiviral vector encoding the human βA T87Q globin gene and stored in cryopreservative solution in the final immediate container for the intended medical use).


Check out the new ITHANET section about Clinical Trials.

More information:

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In an effort to improve our services to the community, we created a new section about clinical trials. The new ITHANET section, "Clinical Trials", is a list of the most recent clinical trials regarding thalassaemia and other haemoglobinopathies. The list is searchable and includes the title, the sponsor's name, the provided keywords and the expected start and completion dates. A link to the corresponding entry is also provided for more detailed information.